ppmd end duchennetour - parent project muscular dystrophy
TRANSCRIPT
Duchenne Patient community
Clinicians
Researchers Industry
Federal Agencies
Congress & Legislators
Health & Regulatory
PolicymakersPayers, PBMs, Valuators, HEs
Coalition Groups
Adults with Duchenne/Becker
Carriers & Manifesting
Carriers
Siblings
PPMD End Duchenne Tour
Parent Project Muscular DystrophyOverview
Kathi Kinnett, MSN APRNSVP Clinical CarePPMD
Research
Advocacy
Care
Education
Grassroots Advocacy
1994 - Questions and Missing Pieces
• What was the current state of research and care in Duchenne?• What was the federal government investment in Duchenne?• What data existed about the Duchenne population? • How do we move the needle toward better care and treatments for
Duchenne?
Care standardsNatural history dataOutcome measures
Federal investment & coordinationCompanies on boardMobilized grassroots community
Results 2001 - Present Care• Care considerations published and updated• Certified Duchenne Care CentersResearch• Wellstone Centers of Excellence• Animal Studies• Basic and Translational Research GrantsData collection • Outcome measures• Natural History Studies• MD-STARnet Surveillance• ICD-10 Code Federal Coordination and funding• MD Coordinating Committee grows • Action Plan for MD’s• 500+million in Duchenne FundingDrug Development & Access• 2 Approved Therapies• 45+ Companies • Pipeline Full of Hope • Pre-competitive, collaborative consortium (PPMD Duchnne Drug Development Roundtable)• Payer Engagement
Public-Private
Partnership
Industry
Patients
Regulators
Clinicians
Federal Agencies
Congress
ANNUAL ADVOCACY CONFERENCE
Federal Agencies We Currently Focus On
For all agenciesWe advocate to impact:
FocusFunding
orFlexibility
PPMD Hill Days Building
Congressional Champions
Appropriations Bill & Annual Report Language
• Decreased heart function• Cardiomyopathy• Leads to heart failure
• Osteoporosis, osteopenia• Risk of fractures
Duchenne affects
more than just muscle
• Constipation• GERD• Stones• Incontinence
• Possible learning, behavior and psychosocial issues (ADD, OCD, speech, learning, behavior, sensory, psychosocial, anxiety, depression)
• Loss of skeletal muscle• Weakness• Decreased function• Decreased ROM
• Weakened diaphragm• Difficulties with airway clearance• Need for assistive ventilation• Risk of pneumonia
Updated Duchenne Care Considerations
Published January 2018• All sections updated to
reflect progress in care & research
• All sections updated to reflect care through the lifespan of Duchenne, not just pediatric care
• New sections within 2018 CDC Care Considerations:
• Primary/Emergency Care
• Endocrine Management
• Transition of Care for Teens & Adults
Care Considerations Pediatrics Supplements Published October 2018
An Introduction to the Updated 2018 Clinical Care Guidance on the Diagnosis and Management of Duchenne
Neurology Care, Diagnostics, and Emerging Therapies of the Patient with Duchenne
Rehabilitation Management of the Patient with Duchenne
Bone Health and Osteoporosis Management of the Patient with Duchenne
Obesity and Endocrine Management of the Patient with Duchenne
Nutritional and Gastrointestinal Management of the Patient with Duchenne
Respiratory Management of the Patient with Duchenne
Cardiac Management of the Patient with Duchenne
Orthopedic and Surgical Management of the Patient with Duchenne
Primary Care and Emergency Department Management of the Patient with Duchenne
Psychosocial Management of the Patient with Duchenne
A Transition Toolkit for Duchenne
Evaluating Implementation of the Updated Care Considerations for Duchenne
Duchenne Family Guide
• Collaborative effort between PPMD, WDO, TREAT NMD and MDA
• Being translated into multiple languages
• Available for download and printing on PPMD website
• Soon to be available in hard copy
Imperatives for DUCHENNE MD
• Collaborative effort between PPMD, WDO and TREAT - NMD
• 1 page snapshot of recommended care
• Helpful for providers and families
• Available on PPMD (English) and on the TREAT NMD site in multiple languages
Certified Duchenne Care Center Program
– Operationalization of the CDC Care Guidelines
– Creating a network that will:
• Provide care in alignment with the Standards of the Certified Duchenne Care Center Program which are in agreement with the CDC Care Considerations
Seattle Children’s Hospital
Stanford Children’s Hospital
Children’s HospitalL.A.
Children’s HospitalColorado
University of Iowa
Children’s Hospital
Cincinnati Children’s Hospital
Nationwide Children’sHospital
DukeUniversity
MedicalCenter
Massachusetts
GeneralHospital
Children’sMercy
Hospital
KennedyKriegerInstitute
UCLA
Yale New Haven
Children’sHospital
LurieChildren’s
HospitalUC Davis
Gillette Children’sHospital
UCSF
AI NemoursDuPont
Children’s National Hospital
American Family
Children’s Hospital
Children’s Hospital
Wisconsin
UVA
Children’s Hospital of the Kings Daughters
U of Rochester
Primary Children’s
Hospital
By April 2019 -
Continuing to Improve the CDCCs• Defining a dashboard and metrics
– New ICD 10 code will enable patient/service identification
– Eventually evaluate longitudinal follow up of patient outcomes
• Updating requirements for certification aligned with updated Care Considerations
• Requirements for Adult Certified Duchenne Care Centers
• Global efforts to standardize care
PPMD Care Resources• PPMD Care Pages
– By stage of disease – By system
• Emergency Card
• Wheelchair Card!
• Emergency checklists
• Mobile app
• FACES Coordinators
• PPMD
Progress and PlansDrug Development RoundtableJanuary 24, 2018Abby Bronson, Joanne Donovan, Cliff Bechtold
Pre-clinical Phase I Phase I/II Phase II Phase IIIExondys 51 (Eteplirsen) [Sarepta]
Emflaza [PTC Therapeutics]Spironolactone & Eplerenone [Ohio State University]
Translarna (Ataluren) [PTC Therapeutics]Givinostat [Italfarmaco]*
Raxone (Idebenone) [Santhera]*SRP-4045/SRP-4053 [Sarepta]*
RG6206 [Roche]*Edasalonexent (CAT-1004) [Catabasis]
Vamorolone (VBP15) [Reveragen]*Ezutromid (SMT C1100) [Summit PLC]*
Pamrevlumab (FG-3019) [Fibrogen]Epicatechin
NS-065/NCNP-01 [NS Pharma]Follistatin Gene Transfer [Nationwide Children's]
MNK-1411 Cosyntropin Acetate [Mallinckrodt]*Myoblast Transplantation [Chu De Quebec]
Exon Skipping 53 [Daichi - Sankyo]***Nationwide Micro-Dystrophin Gene Transfer…
WVE-210201 Exon 51 Skipping [WAVE]Nationwide GalGT2 Gene Therapy [Nationwide…
PF-06939926 Mini-Dystrophin Gene Therapy [Pfizer]SGT-001 Micro-Dystrophin Gene Transfer [Solid]
Rimeporide [EspeRare]**AT-300 [Akashi]
Nationwide Exon 2 Skipping for Duplication 2…Tamoxifen** [University of Geneva]
MA-0211/MTB-1 [Mitobridge/Astellas]
Access &Reimbursement
Our Path to Access?
Photo credit:Conrad Reynoldson
Goals
Provide education and guidance on access process
Provide product specific resources for Patients, Clinicians, Payers
PPMD Duchenne Newborn Screening Program
Duchenne NBS Steering Committee
Outreach & Education to
Patients & HCPs WG
Care Considerations for Infants with
DMD WG
Laboratory Test Refinement WG
NBSTRN Integration and LPDR WG
Ethical & Legal Considerations WG
Evidence Review WG
• PPMD Convened Meeting of Stakeholders to Discuss Model of Pilot: October 2017; Pilot launched October 2018
• Complete CK Assay Validation Studies: FDA approval of PKI kit expected Q1 2019
Contact [email protected]
State Specific Resources –22 PPMD Connect Groups & Growing
Resource Categories
Tracking payer determinations by state & payer and integrating them into our website…
What Can YOU Do ?!• Advocate!! Washington DC!!!
• RUN!!!
Coach to Cure MD!!
• Join a Connect Group!